Nonprofit foundations spur translational research

Abstract

Every year, hundreds of promising basic discoveries in the pharmacological field are lost and will never have a chance to be exploited for patients due to difficulties in clinical translation. This is especially true for most neurodegenerative disorders lacking disease-modifying therapies. Here we present the current scenario and our vision of a 'collective-impact' multistakeholder approach to expedite the development of new drugs.

An “all-wheel drive” proposal to accelerate clinical research in common and rare neurological diseases

“…In the UK, the National MS Society is supporting an expert consortium that has the objective to design and deliver an efficient clinical trials platform to speed up the development of new treatments for progressive MS. We here propose a similar initiative but transversal to different neurological diseases. As already suggested [3], this may represent a step forward: developing therapies across biologically similar diseases may refine our ability to capture the biological efficacy of a pharmacological agent. Consequently, it may de-risk industrial investments, provide results that are perceived as more reliable by regulatory authorities and help develop the economies of scales that may promote the industrialisation of rare disease therapy development.…”

“…During the past 10 years, the international role of patient-advocacy organizations in health research has drastically changed. In Italy, some of them are no longer merely distributors of grant money but have reinvented themselves as on-profit enterprises with new, creative business models [3]. This shift might also reflect a change in the staffing of medical nonprofit foundations from grant managers to experienced pharma and biotech executives.…”

“…Others and we have recently proposed models to improve the collaboration between the key stakeholders in the arena: academia, industry and patient-advocacy organizations [2][3][4]. We suggested that, based on the growing genetic and neurobiological evidence for pathophysiologies that are shared in common across numerous neurological conditions [4], coordinated clinical research initiatives referring to conditions that share biological mechanisms should be encouraged.…”

Progressive MS Alliance Industry Forum: Maximizing Collective Impact To Enable Drug Development

“…Progressive MS Alliance Industry Forum Challenge Moving forward to fulfill the mission of the Progressive MS Alliance we should embrace the unique opportunity to fully advance the science of multi-stakeholder collaborations [16]. In this case, collective impact is a much broader concept than how research outcomes have traditionally been measured.…”

Steps towards Collective Sustainability in Biomedical Research

“…Despite the reasons to be optimistic, including the flow of resources and the different models that exist for collaboration, some concern is emerging about the ability of these partnerships to evolve in a coordinated and productive manner and whether these partnerships can match patients' needs [1,2]. In the face of these concerns, suggestions have been put forward, ranging from models of collaboration in specific diseases [3] to measures to improve the impact of philanthropy [4].…”